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Pediatr Transplant. 2011 Mar;15(2):161-6. doi: 10.1111/j.1399-3046.2010.01431.x. Epub 2010 Dec 22.

Reduced-intensity conditioning for alternative donor hematopoietic stem cell transplantation in patients with dyskeratosis congenita.

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Department of Pediatrics, Nagoya University Graduate School of Medicine, Clinical Research Center for Blood Diseases, National Hospital Organization Nagoya Medical Center, Nagoya, Japan.


DC is an inherited bone marrow failure syndrome mainly characterized by nail dystrophy, abnormal skin pigmentation, and oral leukoplakia. Bone marrow failure is the most common cause of death in patients with DC. Because previous results of HSCT with a myeloablative regimen were disappointing, we used a reduced-intensity conditioning regimen for two patients with classic DC, and one patient with cryptic DC who harbored the TERT mutation. Graft sources included two mismatched-related bone marrow (BM) donors and one unrelated BM donor. Successful engraftment was achieved with few regimen-related toxicities in all patients. They were alive 10, 66, and 72 months after transplantation, respectively. Long-term follow-up is crucial to determine the late effects of our conditioning regimen.

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